RWE Usage

[Danielle]

Can you clarify what parameters to keep in mind when it comes to scales used for efficacy endpoints in RWE studies vs. in the pivotal trials. Ex. Is there an issue if pivotal studies use EGSS and RWE uses IGA?

[Jennifer Carroll]

Hi @Danielle

Section 1.1.4 Endpoints/ Outcomes of the Guidance on Real-World Evidence states “Endpoints/Outcomes must be “consistent with” (though not necessarily “the same as”) those in the TMA. Regardless of whether the evidentiary basis for the presentation is RWE/RWD or an RCT, endpoints are not generally limited to those explicitly included within the TMA. Though the approach for RWE/RWD mirrors that for RCTs in this respect, the following examples are intended to clarify questions received during the consultation process.” You can review the examples in the linked document. Assessment of “consistent with” is made in the context of the combination of the endpoint, TMA and the therapeutic area. The submitter should provide authoritative support within the submission, to support that the endpoint is consistent or similar to those in the TMA and that it is a recognized endpoint within the therapeutic area.